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Appendix B Speaker Biographical Sketches Christopher P. Austin, M.D., is scientific director at the National Institutes of Health (NIH) Center for Translational Therapeutics (NCTT), and direc- tor of the Division of Preclinical Innovation at the National Center for Advancing Translational Sciences, U.S. NIH. The NCTT's mission is both to translate basic science discoveries into new treatments, particularly for rare and neglected diseases, and to develop new technologies and para- digms by which therapeutic development is done. The NCTT's programs span the spectrum of translational science from RNAi biology to small molecule probe discovery to drug development, including genome-wide RNAi, the NIH Chemical Genomics Center (NCGC), the Therapeutics for Rare and Neglected Diseases (TRND) program, and the Bridging Interven- tional Development Gaps (formerly the NIH-RAID) program. The NCGC is an ultra-high-throughput screening, informatics, and chemistry center that profiles small molecule libraries for biological activity using its quan- titative high-throughput screening (qHTS) technology and develops novel compounds as probes of biology and starting points for the development of new drugs for rare and neglected diseases. The NCGC is a partner with the National Toxicology Project, the Environmental Protection Agency, and the Food and Drug Administration in the Toxicology in the 21st Century Pro- gram, which is developing in vitro signatures for in vivo toxicity endpoints. The TRND program develops small molecules and biologics from lead to clinical proof of concept for rare and neglected diseases. Before joining NIH in 2002, Dr. Austin directed research programs in genomics-based target discovery, pharmacogenomics, and neuro psychiatric drug development at Merck, with a particular focus on schizophrenia. Dr. Austin received his 65
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66 GENOME-BASED THERAPEUTICS A.B. in biology summa cum laude from Princeton and his M.D. from Har- vard Medical School. He completed clinical training in internal medicine and neurology at the Massachusetts General Hospital and finished a post- doctoral fellowship in genetics at Harvard. Wylie Burke, M.D., Ph.D., is professor and chair of the Department of Bioethics and Humanities at the University of Washington. She received a Ph.D. in genetics and an M.D. from the University of Washington and completed a residency in internal medicine at the University of Washington. She was a medical genetics fellow at the University of Washington from 1981 to 1982. Dr. Burke was a member of the Department of Medicine at the University of Washington from 1983 to 2000, where she served as associate director of the internal medicine residency program and founding director of the University of Washington's Women's Health Care Center. She was appointed chair of the Department of Medical History (now the Department of Bioethics and Humanities) in October 2000. She is also an adjunct professor of medicine and epidemiology and a member of the Fred Hutchinson Cancer Research Center. She is a member of the Institute of Medicine and the Association of American Physicians and is a past president of the American Society of Human Genetics. Dr. Burke's research addresses the social, ethical, and policy implications of genetics, including responsible conduct of genetic and genomic research, genetic test evalua- tion, and implications of genomic health care for underserved populations. She is director of the University of Washington Center for Genomics and Healthcare Equality, a National Human Genome Research Institute center of excellence in ethical, legal, and social implications research, and co- director of the Northwest-Alaska Pharmacogenomic Research Network. Walter Capone, M.B.A., is the chief operating officer of the Multiple Myeloma Research Foundation (MMRF) and the Multiple Myeloma Research Consor- tium (MMRC). He oversees the core business operations of the MMRF and as part of the executive committee executes the growth initiatives outlined in the organization's strategic plan. He has 20 years of pharmaceutical and biotechnology leadership experience in the areas of commercial develop- ment, operations, finance, marketing, and sales in the United States and internationally. Prior to joining the MMRF, he was the vice president of commercial development and operations at Progenics Pharmaceuticals. He previously worked at a number of entrepreneurial pharmaceutical and bio- technology ventures throughout the United States and Europe including Trimeris, T riangle Pharmaceuticals, and Cyanamid Benelux. He started his career at leading global pharmaceutical companies including Lederle, Wyeth, and Bristol-Myers Squibb. He received his B.A. in international relations
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APPENDIX B 67 from Brown University and he has an M.B.A. in finance and international business from Columbia University Business School. Nicholas Davies, Ph.D., is a partner in the pharmaceutical and life sciences practice at PricewaterhouseCoopers (PwC), responsible for pharmaceuti- cal and research and development strategy. He has been a key figure in the pharmaceutical life sciences consulting and pharmaceutical industry for over 16 years and possesses extensive knowledge spanning research, development, commercial and marketing, and the external payer, access, and regulator environments. Prior to joining PwC, Dr. Davies was respon- sible for global research and development strategy at Pfizer and led a number of key research and development transformations, integrations, and mergers. As a former PwC management consultant and IBM business consulting research and development leader, he led and delivered complex engagements in major mergers and acquisitions, extensive restructuring and downsizing, research and development productivity projects, personalized medicine and diagnostics strategies, portfolio and decision making strate- gies, outsourcing, clinical research organization and partnering strategies, operational strategies in discovery and clinical development, enterprise risk management, Asia and emerging market strategies, out- and in-licensing, and commercial assessments of pipeline assets. He has also led research laboratories and departments at Novartis and AstraZeneca. Dr. Davies gained his Ph.D. in immunology and genetics at Cambridge University, UK. Radoje Drmanac, Ph.D., is a co-founder of Complete Genomics and has served as chief scientific officer since July 2005. In 2001, Dr. Drmanac co- founded Callida Genomics, Inc., a DNA sequencing company, and served as Callida's chief scientific officer from 2001 to 2004 and as its president since 2004. In 1994, Dr. Drmanac co-founded Hyseq, Inc., a DNA array technol- ogy company that became Hyseq Pharmaceuticals, Inc. and later merged with Variagenics, Inc. to become Nuvelo, Inc., and served as its senior vice president of research from 1994 to 1998 and as its chief scientific officer from 1998 to 2001. Prior to that, Dr. Drmanac served as a group leader at Argonne National Laboratory. Dr. Drmanac received a B.S., M.S., and Ph.D. in molecular biology from the University of Belgrade. Deborah Dunsire, M.D., has been the president and chief executive officer of Millennium Pharmaceuticals Inc., since July 2005. In 2008 Millennium was acquired by Takeda Pharmaceuticals of Japan, becoming the global oncol- ogy center of excellence for Takeda under her leadership. Dr. Dunsire joined Millennium from her role as senior vice president and North A merican region head of the oncology business unit of Novartis. Prior to her move to the United States in 1994, Dr. Dunsire worked in the global headquarters
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68 GENOME-BASED THERAPEUTICS of Sandoz in Switzerland managing launch and growth of global products in the field of immunology and dermatology. She joined Sandoz in South Africa in 1988 as a clinical researcher and expanded her résumé to include portfolio and specialty market management. Dr. Dunsire is a member of the board of directors of Allergan Inc. and serves as a director of the Bio- technology Industry Organization where she co-chairs the Committee on Reimbursement and serves as a member of the executive committee. Her not-for-profit board memberships include CancerCare Inc.; the Gabrielle's Angel Foundation for Cancer Research; and the Boston Museum of Science, where she chairs the investment review board; and she is also a member of the Massachusetts General Hospital research advisory council. She served as a director of the Pharmaceutical Research and Manufacturers of America from 2005 to 2008 and as a director of the California Healthcare Institute from 2002 to 2005. Dr. Dunsire was the 2001 recipient of the American Cancer Society Excalibur Award, the 2000 recipient of the Health Care Busi- ness Women's Association Rising Star Award, the 2009 Health Care Business Women's Association Woman of the Year Award, the 2011 Massachusetts Biotechnology Organization's Innovative Leadership Award, and the 2011 Golden Door Award from the International Institute of New England. She received a Ph.D. honoris causa from Worcester Polytechnic Institute in 2007. Dr. Dunsire graduated as a physician from the University of Witwatersrand in Johannesburg, South Africa. Victor J. Dzau, M.D., is the chancellor for health affairs and James B. Duke Professor of Medicine at Duke University and the president and chief execu- tive officer of Duke University Health System. Dr. Dzau was previously the Hersey Professor of Theory and Practice of Medicine and chairman of medicine at Harvard Medical School's Brigham and Women's Hospital and the chairman of the department of medicine at Stanford University. Dr. Dzau has made a significant impact on medicine through his seminal research in cardiovascular medicine, his pioneering work in the discipline of vascular medicine, and recently his leadership in health care innovation. His important work on the renin angiotensin system (RAS) paved the way for the contemporary understanding of RAS in cardiovascular disease and the development of RAS inhibitors as therapeutics. Dr. Dzau also pioneered gene therapy for vascular disease and was the first to introduce DNA decoy molecules to block transcription as gene therapy in vivo. Recently his semi- nal work on stem cell "paracrine mechanism" provided novel insights into stem cell biology and therapy. As a leader of academic medicine, Dr. Dzau's vision is that academic health centers must lead the transformation through innovation, translation, and globalization. To achieve this vision he has established the Duke Translational Medicine Institute, Duke Global Health Institute, Duke Initiative in Health Innovation, and Duke National Univer-
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APPENDIX B 69 sity of Singapore Graduate Medical School in Singapore. Among his honors and recognitions are the prestigious Gustav Nylin Medal from the S wedish Royal College of Medicine; the Max Delbruck Medal from Humboldt Uni- versity, Charite and Max Planck Institute; the Commemorative Gold Medal from Ludwig Maximillian University of Munich and Frey-Werle Founda- tion; the Inaugural Hatter Award from the Medical Research Council of South Africa; the Polzer Prize from the European Society of Sciences and Arts; the Ellis Island Medal of Honor of the USA; the Novartis Award for Hypertension Research; the Distinguished Scientist Award from the Ameri- can Heart Association (AHA); and the 2010 AHA Research Achievement Award for his contributions to cardiovascular biology and medicine. He has received six honorary doctorates. He serves on the council of the Institute of Medicine of the National Academy of Sciences, the board of directors of Research America, and the board of health governors of the World Economic Forum. He is also board chair of the Association of Academic Health Centers. He has chaired the NIH Cardiovascular Disease Advisory Committee as well as the Council of Atherosclerosis, Thrombosis and Vas- cular Biology of the AHA, and has served on the advisory council to the director of NIH. Garret A. FitzGerald, M.D., FAHA, is the McNeil Professor in Translational Medicine and Therapeutics at the University of Pennsylvania in Philadelphia, where he chairs the Department of Pharmacology and directs the Insti- tute for Translational Medicine and Therapeutics (ITMAT). Dr. FitzGerald trained in medicine at University College Dublin and its teaching hospitals and in statistics at Trinity College in Dublin and the London School of Hygiene. Following fellowships at the Royal Postgraduate Medical School in London, the Max Planck Institute in Cologne, and Vanderbilt University, Dr. FitzGerald joined the faculty at Vanderbilt and eventually led the Divi- sion of Clinical Pharmacology as the William Stokes Professor of Experi- mental Therapeutics. He moved in 1991 to lead the Department of Medicine and Experimental Therapeutics at University College, Dublin, and then returned in 1994 to the United States to take up direction of the Center for Experimental Therapeutics and the General Clinical Research Center as the Robinette Professor of Cardiovascular Medicine at Penn. These struc- tures were subsumed into ITMAT when it was founded in 2004, antici- pating the funding of clinical and translational research centers 2 years later by the NIH. ITMAT has grown to more than 650 members and supports research programs, faculty recruitment, education, and infrastructural devel- opments relevant to translational research. Dr. FitzGerald has served as chair of the Department of Pharmacology at Penn. The department is routinely placed in the top three in NIH funding in the United States and supports a graduate group in pharmacological sciences with about 90 students.
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70 GENOME-BASED THERAPEUTICS Dr. FitzGerald's research has been characterized by an integrative approach to elucidating the mechanisms of drug action, drawing on work in cells, model organisms, and humans. His work contributed substantially to the development of low-dose aspirin. He was the first to describe the dose- dependent suppression by aspirin of thromboxane and p rostacyclin bio synthesis in vivo and to discover that inhibition of platelet c yclooxygenase by low-dose aspirin occurred in the presystemic circulation and to char- acterize the interaction by which non-steroidal anti- inflammatory drugs (NSAIDs) like ibuprofen could interact with and undermine cardio protection from aspirin. Dr. FitzGerald's group was the first to predict and then mechanistically explain the cardiovascular hazard from NSAIDs. Since his first prediction of a potential hazard, based on clinical pharma- cological studies 12 years ago, evidence consistent with the mechanism proposed-- suppression of COX-2 derived prostacyclin--has emerged from multiple studies in model systems, including many genetically manipu- lated mice created by his group; human genetics; randomized comparisons amongst NSAIDs; and seven placebo-controlled trials of three structurally distinct NSAIDs designed to be specific for inhibition of COX-2. Aside from this work, Dr. FitzGerald has also discovered many products of lipid peroxidation and established their utility as indices of oxidant stress in vivo. Using this methodology he demonstrated that conventional doses of vitamins E and C have no impact on lipid peroxidation in healthy individu- als with intact endogenous antioxidant defense, that social consumption of alcohol has a pro-oxidant effect, and that suppression of lipid peroxidation retards atherogenesis in mice. His laboratory was the first to discover a molecular clock in the cardiovascular system and has contributed substan- tially to our understanding of the importance of peripheral clocks in the regulation of cardiovascular and metabolic function. Dr. FitzGerald's papers have been published in journals such as Cell, Science, Nature, the New England Journal of Medicine, the Lancet, JAMA, PNAS, JCI, and Nature Medicine and have been cited more than 30,000 times. He has also published on science policy in the lay and profes- sional press and is an occasional sports commentator for Il Manifesto. He has been awarded honorary degrees from University College Dublin and the Universities of Edinburgh and Frankfurt. Among his awards are the Harvey Medal, the Boyle Medal, the Taylor Prize, and the Cameron Prize. Dr. FitzGerald serves on the peer review advisory committee of the NIH, the science board of the FDA, and the drug forum of the Institute of Medicine. Jane Fridlyand, Ph.D., is a senior statistical scientist in the Department of Biostatistics at Genentech/Roche. Dr. Fridlyand received her Ph.D. in statistics in 2001 from the University of California, Berkeley; her disser- tation was focused on the applications of statistics to high-dimensional
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APPENDIX B 71 biological data, including sequencing, genotyping, and early expression microarrays. She continued on to a postdoctoral position at the University of California, San Francisco (UCSF) Cancer Center where she developed novel methods for the analyses of genome-wide copy-number data. In 2003 she transitioned to a faculty position at the Department of Epidemiology and Biostatistics at UCSF. Dr. Fridlyand's main area of research included development of new approaches to the integration of different modalities of high-dimensional genomic and genetic data in cancer with the aim of identifying novel tumor subtypes relevant to disease etiology and prognosis. Dr. Fridlyand has co-authored more than 50 peer-reviewed publications and multiple book chapters, was a key contributor to a number of funded NIH applications, and has been an invited speaker at many national and international meetings. In 2007, Dr. Fridlyand joined early clinical develop- ment, oncology, at Genentech. In the past 5 years, her work has focused on developing strategies for incorporation of biomarkers into clinical develop- ment programs. Currently she leads global biometrics efforts at Roche in personalized health care. Felix W. Frueh, Ph.D., is president of the Medco Research Institute, lead- ing Medco's real-world, outcomes-based research in personalized medicine. Dr. Frueh was associate director for genomics at the FDA and managing partner at Stepoutside Consulting and held senior positions at Transgenomic and Protogene Laboratories. He is a member of the board of the Person- alized Medicine Coalition and TcLand Expression, Inc. and is adjunct faculty at the Institute for Pharmacogenomics and Individualized Therapy at the University of North Carolina. Dr. Frueh held faculty appointments in the departments of pharmacology and medicine at Georgetown University in Washington, DC, and was a fellow at Stanford University and the Univer- sity of Basel, Switzerland, where he also received his Ph.D. in biochemistry. Geoffrey Ginsburg, M.D., Ph.D., is the founding director for Genomic Medicine at Duke University and assumed his current position in the Duke Institute for Genome Sciences & Policy in 2004. He is also the founding executive director of the Center for Personalized Medicine established in the Duke University Health System in 2010. Dr. Ginsburg is currently professor of medicine and pathology at Duke University Medical Center. While at Duke, Dr. Ginsburg has pioneered translational genomics, initiating pro- grams in genome enabled biomarker discovery, longitudinal registries with linked molecular and clinical data, biomarker-informed clinical trials, and the development of novel practice models and implementation research for the integration of genomic tools in heath care systems. With a strong com- mitment to interdisciplinary science he has led projects to develop predictive models for common complex diseases using high-dimensional genomic data
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72 GENOME-BASED THERAPEUTICS as well as collaborations with engineering groups to develop novel point of care sensors. Dr. Ginsburg's work spans oncology, infectious diseases, cardiovascular disease and metabolic disorders. His research is addressing the challenges for translating genomic information into medical practice using new and innovative paradigms and the integration of personalized medicine into health care. He is an internationally recognized expert in genomics and personalized medicine with over 200 published papers and funding from NIH, DOD, DARPA, the Gates Foundation, and industry. In 1990, he joined the faculty of Harvard Medical School, where he was director of Preventive Cardiology at Beth Israel Hospital and led a laboratory in applied genetics of cardiovascular disease at Children's Hospital. In 1997 he joined M illennium Pharmaceuticals Inc. as senior program director for cardiovascular diseases and was eventually appointed vice president of Molecular and Personalized Medicine, where he was responsible for devel- oping pharmacogenomic strategies for therapeutics, as well as biomarkers for disease and their implementation in the drug development process. He has received a number of awards for his research accomplishments, includ- ing the Innovator in Medicine Award from Millennium in 2004 and the Basic Research Achievement Award in Cardiovascular Medicine from Duke University in 2005. He is a founding member and former board member of the Personalized Medicine Coalition, a senior consulting editor for the Journal of the American College of Cardiology, an editor for the HUGO Journal, and an editorial advisor for Science Translational Medicine. In addition he is the editor of Genomic and Personalized Medicine (Elsevier) whose first edition was published in 2009. Dr. Ginsburg has been a member of the Secretary of Veterans Affairs Advisory Council on Genomic Medicine and the National Advisory Coun- cil for Human Genome Research at NIH. He is currently an international expert panel member for Genome Canada, a member of the Board of External Experts for the National Heart, Lung, and Blood Institute, the Institute of Medicine's Roundtable on Translating Genomic-Based Research for Health, and a member of the External Scientific Panel for the Pharmaco genomics Research Network. Dr. Ginsburg has recently been appointed to the Advisory Council for the newly established National Center for Advanc- ing Translational Sciences at NIH and has recently been nominated to serve on the World Economics Forum's Global Agenda Council on Personalized and Precision Medicine. Dr. Ginsburg received his M.D. and Ph.D. in biophysics from Boston University and completed an internal medicine residency at Beth Israel Hospital in Boston, Massachusetts. Subsequently, he pursued postdoctoral training in clinical cardiovascular medicine at Beth Israel Hospital and in molecular biology at Children's Hospital as a Bugher Foundation Fellow of the American Heart Association.
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APPENDIX B 73 Margaret A. Hamburg, M.D., is the 21st commissioner of the U.S. Food and Drug Administration (FDA). As the top official at the FDA, Dr. Ham- burg is committed to strengthening programs and policies that enable the agency to carry out its fundamental mission--to protect and promote the public health. Only the second woman ever to serve as commissioner, Dr. Hamburg earned her M.D. from Harvard Medical School and com- pleted her residency at what is now New York Presbyterian HospitalWeill Cornell Medical Center. She conducted neuroscience research at Rockefeller University in New York and at the National Institute of Mental Health and later focused on AIDS research as assistant director of the National Institute of Allergy and Infectious Diseases. In 1991, after just a year in the New York City Department of Health, Dr. Hamburg was named its com- missioner. During her 6-year tenure she implemented rigorous public health initiatives that tackled the city's most pressing crises head-on, including improved services for women and children, a needle-exchange program to combat HIV transmission, and the nation's first public health bioterrorism defense program. The most celebrated achievement during her leadership was her aggressive approach to the city's tuberculosis epidemic, which led to an 86 percent decline in drug-resistant TB in just 5 years. In 1997, 3 years after she was elected one of the youngest-ever members of the Institute of Medicine, President Bill Clinton named Dr. Hamburg assistant secretary for planning and evaluation in the U.S. Department of Health and Human Services, where she served until the end of the Clinton administration. She then became founding vice president for biological programs at the Nuclear Threat Initiative, a foundation dedicated to reducing the threat to public safety from nuclear, chemical, and biological weapons. President Barack Obama nominated Dr. Hamburg for the post of FDA commissioner on March 14, 2009. As the commissioner of food and drugs, Dr. Hamburg has emphasized the critical role of innovation in meeting the nation's rapidly growing public health needs. She provided leadership for the implementa- tion of three groundbreaking measures: the Family Smoking Prevention and Tobacco Control Act, a 2009 law that gives FDA the authority to regulate the manufacture, distribution, and marketing of tobacco products; the Food Safety Modernization Act of 2011, which changed the focus of food safety measures from responding to food-borne outbreaks of illness to preventing them; and a thorough review of the system for the evaluation and approval of medical devices. Beyond these specific undertakings, Dr. Hamburg has set the agency's paramount course for fulfilling two central public health tasks. She has launched a nationwide public-private effort to strengthen regula- tory science as a means for advancing the development and evaluation of innovative, breakthrough medical products, and she is leading FDA's trans- formation into a global regulatory agency capable of ensuring the safety and quality of imported food, drugs, and medical devices. Commissioner
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74 GENOME-BASED THERAPEUTICS Hamburg is committed to ensuring that FDA is poised to meet the public health challenges of the 21st century. Steffan N. Ho, M.D., Ph.D., is currently director of translational oncol- ogy at Pfizer Inc. He received a Ph.D. in immunology and an M.D. from the Mayo Clinic. He completed a residency in pathology at the Stanford University Medical Center and a postdoctoral fellowship in the Howard Hughes Medical Institute, also at Stanford. Dr. Ho was on the faculty at the University of California, San Diego, School of Medicine in the Department of Pathology and the Department of Cellular and Molecular Medicine. He subsequently led the development of the translational oncology group at Biogen Idec. In his current position at Pfizer, Dr. Ho plays a leadership role in integrating translational research efforts with experimental medicine strategies to define mechanism of action, understand pharmacodynamic relationships, identify rational therapeutic combinations, and investigate predictive biomarker hypotheses. He has focused on integrating drug and diagnostic development strategies, including the coordination of strategic collaborations supporting predictive diagnostic test development. He also functions as the translational oncology lead for the Xalkori program. Kathy L. Hudson, Ph.D., is the deputy director for science, outreach, and policy at the National Institutes of Health (NIH) where she oversees the activities of the associate directors for communications and public liaison, legislative policy and analysis, and science policy. In addition, Dr. Hudson works with NIH leadership to develop and implement new strategic and scientific initiatives and is the NIH liaison with the U.S. Department of Health and Human Services. She also represents the NIH--and the NIH director--in high-level collaborations and negotiations with other federal agencies, such as FDA, Centers for Disease Control and Prevention, and the White House Office of Science and Technology Policy, as well as with private research institutions, patient voluntary organizations, and profes- sional societies. In addition to her role in the Office of the Director at NIH, in December 2011 Dr. Hudson became the acting deputy director of the new National Center for Advancing Translational Sciences (NCATS) at NIH. She also serves as the acting director of the Office of Strategic Communications, Alliances, and Policy at NCATS. Dr. Hudson holds a Ph.D. in molecular biology from the University of California, Berkeley, an M.S. in microbiology from the University of Chicago, and a B.A. in biology from Carleton College. Thomas Lehner, Ph.D., M.P.H., is the director of the Office for G enomics Research Coordination and chief, Genomics Research Branch at the National Institute of Mental Health (NIMH), National Institutes of Health
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APPENDIX B 75 (NIH). He oversees and coordinates all efforts associated with genomics research for NIMH and is the principal advisor to the NIMH director and the NIMH scientific director for issues related to genetics and genom- ics. A native of Vienna, Austria, he received a Ph.D. in genetics from the University of Vienna and an M.P.H. in epidemiology from Columbia University. He completed his doctoral training in the laboratory of Jurg Ott at Columbia University and later moved to the Rockefeller University where he worked with Jeff Friedman as the Associate Director of the Starr Center for Human Genetics and as senior research scientist affiliated with the Laboratory for Statistical Genetics. He has also served as an infectious disease epidemiologist for the City of New York and as director of science and research for a subsidiary of Millennium Pharmaceuticals. Since joining NIMH in 2004, Thomas has been instrumental in expanding the NIMH genomics portfolio and the NIMH Repository at Rutgers University while also promoting the team science approach in genomics by forging interna- tional collaborative efforts and consortia. Peter Mueller, Ph.D., joined Vertex in July 2003. As executive vice presi- dent, global research and development, and chief scientific officer, he pro- vides strategic oversight for Vertex's worldwide drug discovery research programs, pharmaceutical development, quality assurance and control, and pharmaceutical operations as well as clinical and nonclinical develop- ment, regulatory, and medical affairs. Key areas of Vertex's research and development are hepatitis C (HepC), cystic fibrosis (CF), immune-mediated inflammatory diseases (IMIDs), cancer, and neurological diseases, which led in 2011 to the successful approval and launch of INCIVEK (HepC), a NDA/MAA submission for KALYDECO (CF) with FDA approval in Janu- ary 2012 and several proof of clinical concept candidates in various disease areas. Prior to coming to Vertex, Dr. Mueller served as senior vice president, research and development, for Boehringer Ingelheim Pharmaceuticals, Inc., where he was responsible for the development of all drug candidates of the company's worldwide portfolio in North and South America, Canada, and Japan, beginning in 1997. He also led research programs in the areas of immunology, inflammation, cardiovascular disease, and gene therapy on a global basis. During his time with Boehringer Ingelheim (BI), Dr. Mueller oversaw the discovery of numerous development candidates, held several positions in basic research, medicinal chemistry, and management in dif- ferent centers of BI worldwide. Dr. Mueller received both an undergradu- ate degree and a Ph.D. in chemistry at the Albert Einstein University of Ulm, Germany, where he also holds a professorship in theoretical organic chemistry. He completed fellowships in quantum pharmacology at Oxford University and in biophysics at Rochester University. Special fields of study are synthetic organic chemistry, computational chemistry (cheminformat-
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76 GENOME-BASED THERAPEUTICS ics and bioinformatics), RNA-biophysics, atherosclerosis research, IMIDs, neurodegenerative diseases, infection, oncology, gene/epigenetic technology, and management strategies. He is a board member of various scientific and political societies, such as the Gesellschaft Deutscher Chemiker and Verband Chemische Industrie; the Royal Society of Chemistry; the U.S.- India Chamber of Commerce Biotech, Pharma & Medical Devices Council; the Industrial Research Institute; RNA-the Society; the Association of Stra- tegic Alliance Professionals; the American Association for the Advancement of Science; and the Harvard Accelerator Fund. Before he left Connecticut to join Vertex, Dr. Mueller was also a member of Governor Roland's Council on Economic Competitiveness and Technology for the State of Connecticut. Laura K. Nisenbaum, Ph.D., is senior research advisor in translational medicine and tailored therapeutics at Eli Lilly and Company. She received a Ph.D. in neuroscience from the University of Pittsburgh in 1991. Prior to this, Dr. Nisenbaum received a Fulbright Scholarship to study neuro science in Cologne, Germany. Before joining Lilly, she completed post- doctoral fellow ships at the National Institute of Mental Health and the University of Tennessee College of Medicine. In addition she was an assis- tant p rofessor in the Department of Physiology and Neurobiology at the University of C onnecticut from 1995 to 1998. Dr. Nisenbaum joined Lilly in 1998 and while there she has made significant contributions to drug dis- covery and development for the treatment of psychiatric and neurological disorders, especially schizophrenia. She has developed and implemented molecular profiling methodologies for novel target validation and bio- marker development. In addition, Dr. Nisenbaum has applied pharmaco genomics across the neuroscience drug development portfolio to help tailor Lilly drugs for the right patient, leading to improved individual patient outcomes. Michael A. Pacanowski, Pharm.D., M.P.H., is a clinical pharmacologist and team leader of the genomics group in the Office of Clinical Phar- macology at the FDA. Dr. Pacanowski received his Pharm.D. from the Philadelphia College of Pharmacy. He then completed clinical training at Bassett Healthcare in Cooperstown, New York, and a clinical research fellow ship in cardio vascular pharmacogenomics at the University of Florida, where he also received his M.P.H. Dr. Pacanowski's expertise is in the area of genetic epidemiology and public health genomics, specifi- cally as related to pharmaco genomic strategies in drug development and utilization. At the FDA, he oversees review of investigational and new drug applications, contributes to regulatory policy development, and conducts research that supports FDA's core public health mission.
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APPENDIX B 77 Gary Palmer, M.D., J.D., M.B.A., M.P.H., is a medical oncologist with a career spanning three decades in oncology, initially as a clinician in both the academic and community settings and then as a biotech industry executive with diagnostic and therapeutic experience. Currently, he is the senior vice president of medical affairs and commercial development at Foundation Medicine. Dr. Palmer joined Foundation Medicine from On-Q-ity, where he was chief medical officer and head of development for DNA repair marker development and circulating tumor cell technology. He also served as vice president of medical affairs at Genomic Health, Inc., where he was instrumental in the commercialization of the Oncotype DX breast cancer assay. Prior to Dr. Palmer's tenure with Genomic Health, he held leadership positions at Kosan Biosciences and Salmedix, Inc. He also spent 5 years at Amgen, Inc., where he was involved in the clinical development and commercialization of Neupogen, Neulasla, and Aranesp. Prior to joining industry, he served as director of the Medical Breast Service at the Univer- sity of California, Davis, Cancer Center and chief of medical oncology at Mercy Health System, Sacramento. Dr. Palmer received a B.A. from Yale University and an M.D. from the Stanford University School of Medicine. He completed his internal medicine training at the Boston City Hospital and his oncology fellowship at the Massachusetts General Hospital. He also holds an M.B.A. from the University of California, Davis, an M.P.H. from the University of California, Los Angeles, and a J.D. from Concord Law School. He is a licensed physician and a member of the State Bar of California. Michelle Ann Penny, Ph.D., is a senior director in the translational medicine group at Eli Lilly and Company. She received her Ph.D. in genetics from the University of Birmingham, UK, in 1993. After a postdoctoral fellow- ship in the virology division at the National Institute of Medical Research, Mill Hill, London, she joined the Imperial College London, Department of Medical and Community Genetics where she was a postdoctoral research scientist until taking a lectureship role in human molecular genetics in 1998 as course leader for two master of science programs in human molecular genetics and molecular genetics with genetic counseling. Her academic research career focused on the study of complex polygenic diseases, particu- larly autoimmune disease and susceptibility to infection. In 2002 Dr. Penny joined the clinical pharmacogenomics group at Pfizer in Sandwich, UK, and moved to New London, Connecticut, in 2006 to take on the role of oncol- ogy molecular medicine lead until 2009, when she moved to Indianapolis to lead the pharmacogenomics work at Eli Lilly and Company. Aidan Power, M.B., B.Ch., M.Sc., M.R.C.Psych., has been vice president and head of PharmaTx Precision Medicine since January 2008. Precision
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78 GENOME-BASED THERAPEUTICS medicine represents a synthesis of all the emerging technologies and opera- tions (computational science, imaging, pharmacogenomics, m etabolomics, proteomics, physiological measurements, and diagnostics) that form the scientific basis of emerging approaches to the development of person- alized medicine. Graduating in medicine from the University College Cork, Ireland, Dr. Power trained as a psychiatrist in England and joined Pfizer in the United Kingdom in 1993, working on the antidepressant Sertraline and the antipsychotic Ziprasidone. In 2002 Dr. Power relocated to Pfizer Global Research and Development Headquarters in New London, Connecticut, where he headed clinical pharmacogenomics. For the last 3 years he has headed up molecular medicine (now PharmaTx Precision Medicine), which has been integrating molecular studies across disease areas as well as developing diagnostics for critical programs in the Pfizer product pipeline. Sharon Terry, M.A., is president and CEO of the Genetic Alliance, a net- work dedicated to improving health through the authentic engagement of communities and individuals. She is the founding CEO of PXE Interna- tional, a research advocacy organization for the genetic condition pseudo- xanthoma elasticum (PXE). Following the diagnosis of their two children with PXE in 1994, Sharon, a former college chaplain, and her husband, Patrick, founded and built a dynamic organization that enables ethical research and policies and provides support and information to members and the public. Along with the other co-inventors of the gene associated with PXE (ABCC6), she holds the patent for the invention, and with the assignment of all rights to PXE International, is its steward. She co-directs a 33-lab research consortium and manages 52 offices worldwide for PXE International. Ms. Terry is also a co-founder of the Genetic Alliance Reg- istry and Biobank (GARB). It is a lay-owned and lay-managed biologic samples and data repository catalyzing translational genomic research on genetic diseases. GARB works in partnership with academia and industry to develop novel diagnostics and therapeutics to better understand and treat these diseases. Ms. Terry is at the forefront of consumer participation in genetics research, services, and policy and serves as a member of many of the major governmental advisory committees on biomedical research, including the Health Information Technology Standards Committee for the Office of the National Coordinator for Health Information Technol- ogy, liaison to the Secretary's Advisory Committee on Heritable Disorders in Newborns and Children and the National Advisory Council for Human Genome Research, NHGRI, NIH. She serves on the boards of the Institute of Medicine's Health Sciences Policy Board, National Coalition for Health Professional Education in Genetics, the Coalition for 21st Century Medi- cine, and the International Rare Disease Research Consortium. She is on
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APPENDIX B 79 the editorial board of Genetic Testing and Biomarkers, Biopreservation and Biobanking, and the Google Health and Rosalind Franklin Society Advisory Boards. Ms. Terry is the chair of the Coalition for Genetic Fairness that was instrumental in the passage of the Genetic Information Nondiscrimination Act and is co-chair of the Institute of Medicine's Roundtable on Translat- ing Genomic-Based Research for Health. In 2005, she received an honor- ary doctorate from Iona College for her work in community engagement; the first Patient Service Award from the University of North Carolina Institute for Pharmacogenomics and Individualized Therapy in 2007; the Research!America Distinguished Organization Advocacy Award in 2009; and the Clinical Research Forum and Foundation's Annual Award for Leadership in Public Advocacy in 2011. She is also an Ashoka Fellow. Ms. Terry is committed to personal transformation as a catalyst for the system change needed to improve health and wellness. Mark Trusheim is a visiting scientist and executive in residence at the Massachusetts Institute of Technology (MIT) Sloan School of Manage- ment. He has been a special government employee for the FDA's Office of the Commissioner and is the founder and president of Co-Bio Consulting, LLC. He holds degrees in chemistry from Stanford University and manage- ment from MIT. Mr. Trusheim's research focuses on regional innovation industry economic clusters and modeling the introduction of new innova- tions in health care, such as stratified medicines, to inform public policy, corporate strategy, and product development programs. He is a former member of the Massachusetts Biotechnology Council's board of directors, which helps its more than 500 members succeed in the state. In 2004 he further served as the interim president of the council, leading its success- ful legislative agenda, its expansion of MassBioEd education programs, and its continued membership growth. Co-Bio Consulting focuses on bio- technology public policy, corporate development, and financing. Co-Bio Consulting helps life sciences firms secure partners and rapidly move their research to market by connecting strategy formation to action. The firm also helps facilitate academic, government, and industry consortia to grow life sciences economic clusters. Clients include established biopharma firms, start-up biotechs, universities, and government agencies. As an entrepre- neur, Mr. Trusheim founded and was the first president and chief execu- tive officer of Cantata Laboratories. Cantata marketed clinical diagnostics and pharmaceutical biomarker services based on its biochemical profiling platform. Prior to Cantata, Mr. Trusheim worked at Monsanto/Pharmacia, culminating his career there as co-president and chief operating officer of Cereon Genomics, LLC. Located in Cambridge, Massachusetts, Cereon was created in 1997 by Monsanto as part of a $500 million collaboration with Millennium Pharmaceuticals. Prior to his position at Cereon, Mr. Trusheim
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80 GENOME-BASED THERAPEUTICS was responsible for Monsanto's external genomics relationships and held roles of vice president in the health and wellness sector, marketing director in Searle Pharmaceutical, and director in the agriculture division strategy. Mr. Trusheim spent the first half of his career in the high-tech industry working at Wang Laboratories in computer hardware and at the startup Kenan Systems Corporation, which focused on developing quantitative models and artificial intelligencebased applications for large corporations and government agencies.